While scientists understand the biochemical nuances of these mutations, their implications on neuronal development and function remain a mystery. To address this puzzle, HSCI Associate Faculty member Stephen Haggarty, PhD, reprogrammed a series of both mutated and non-mutated cells back into a stem cell state in which they have the ability to derive new tissues.

Haggarty and his team found that the FMR1 mutations present in the induced pluripotent stem cells (iPSCs) do not always resemble those in the naturally occurring cells from which they came. This offers valuable information as other researchers begin to design investigations using these iPSCs.

Additionally, the team used the cell lines to generate a variety of neuronal cell types. While FMRP loss did not prevent neurodevelopment, it did impact cell quality, suggesting an important role for FMRP early in human neurodevelopment. These findings will allow researchers to characterize existing drugs and develop new therapies for the treatment of some ASDs.

Stem cells are basic cells that can become almost any type of cell in the body. Human stem cells can come from an embryo or an adult human. They have many possible uses in science and medicine, yet controversy surrounds them.

New research has identified rogue cells -- namely brain and muscle cells -- lurking within kidney organoids. Such cells make up only 10 to 20 percent of an organoid's cells, but their presence indicates that the 'recipes' used to coax stem cells into becoming kidney cells inadvertently are churning out other cell types.

A new study shows that cancer stem cells switch from metabolizing sugar to metabolizing protein. Clinical trial based on this observation may revolutionize care for older adults with acute myeloid leukemia.

Researchers have developed a way to grow human heart tissue that can serve as a model for the upper chambers of the heart, known as the atria. The tissue, derived from human induced pluripotent stem cells (hiPCSs), beats, expresses genes, and responds to drugs in a manner similar to a real human atrium. The model […]

Scientists have created a tiny, biodegradable scaffold to transplant stem cells and deliver drugs, which may help treat Alzheimer's and Parkinson's diseases, aging brain degeneration, spinal cord injuries and traumatic brain injuries. Stem cell transplantation, which shows promise as a treatment for central nervous system diseases, has been hampered by low cell survival rates, incomplete […]

Promising findings from preclinical animal studies show the potential of gene therapy for treating incurable neurological disorders. Scientists have successfully used gene therapy to slow the progression and improve symptoms of disorders such as amyotrophic lateral sclerosis and Parkinson's disease.

A new form of therapy may halt or even reverse a form of progressive vision loss that, until now, has inevitably led to blindness. This hyper-targeted approach offers hope to individuals living with spinocerebellar ataxia type 7 (SCA7) and validates a new form of therapy with the potential to treat neurogenetic diseases effectively and with […]